BrainChild Bio advances CAR-T therapy for pediatric brain tumors

BrainChild Bio will advance its lead autologous CAR-T cell therapy candidate for a type of incurable pediatric brain tumor into a phase 2 pivotal clinical trial.

Following a Type B meeting with the U.S. FDA, BrainChild aligned with the regulator on a clinical plan to advance BCB-276, a CAR-T cell therapy targeting the immune checkpoint B7-H3, directly into a single pivotal registration trial designed to accelerate the path to submit a BLA for the treatment of children and young adults with intrinsic pontine glioma (DIPG).

This potentially accelerated clinical path for BCB-276 is supported by the promising preliminary safety and efficacy data for SCRI-CARB7H3(s) — the research cell product that derived BCB-276 — from a phase 1 clinical trial conducted by Seattle Children’s. The clinical data from a subset of 21 DIPG patients from the phase 1 study included 12 patients who began their CAR-T treatment after disease progression and 9 patients who began treatment before disease progression. Preliminary efficacy analyses demonstrate a median time from diagnosis to death for all 21 patients treated was 19.8 months. Three patients, all of which began CAR-T treatment prior to disease progression, remained alive at 44.6 months, 45.6 months and 52.5 months from diagnosis.

DIPG is a fatal brainstem tumor that affects 200-300 children per year in the U.S. Current standard-of-care treatment remains limited to palliative focal radiation therapy which results in a median overall survival of only 8-11 months from diagnosis.